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Rare disease UK

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FDA approves Geron’s Rytelo to treat lower-risk myelodysplastic syndrome patients

The oligonucleotide telomerase inhibitor is administered as an intravenous infusion every four weeks

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Takeda’s enzyme replacement therapy recommended by CHMP for rare blood clotting disorder cTTP

If approved, rADAMTS13 will be the first and only enzyme replacement therapy in the EU indicated for cTTP

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Bristol Myers Squibb’s Breyanzi receives FDA approval for mantle cell lymphoma

The rare form of non-Hodgkin lymphoma accounts for around 3% of all NHL cases

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Moderna’s RSV vaccine mRESVIA granted FDA approval for use in older adults

The approval marks the first time an mRNA vaccine has been approved for a disease other than COVID-19

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Takeda and Degron enter molecular glue degrader partnership worth over $1.2bn

The companies will focus on targets in oncology, neuroscience and inflammation

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Takeda’s Qdenga becomes second dengue vaccine to receive WHO prequalification

Approximately half of the global population is at risk of mosquito-borne viral disease

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Takeda gains exclusive option for AC Immune’s Alzheimer’s drug candidate in deal worth over $2.1bn

The agreement includes an option to licence global rights to the anti-amyloid beta immunotherapy

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Takeda’s fruquintinib recommended by CHMP for metastatic colorectal cancer

Approximately 520,000 new cases of colorectal cancer were diagnosed in Europe in 2020

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Takeda’s subcutaneous Entyvio approved by FDA as Crohn’s disease maintenance therapy

Inflammatory bowel disease affects around ten million people worldwide

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Takeda gains rights to Kumquat’s immune-oncology programme in deal worth over $1.2bn

The selected candidate can be developed as a monotherapy, combination therapy or both

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Takeda’s Takhzyro granted MHRA approval for younger hereditary angioedema patients

The rare genetic disorder is estimated to affect one in every 50,000 people

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Takeda’s HyQvia receives MHRA approval for rare neurological disorder CIDP

The therapy offers patients with CIDP a typical dosing interval of four weeks

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